Myotonic Dystrophy, Type 1 (DM1)Phase 1NCT03959189
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
A Phase 1 study testing ERX-963 in people with Myotonic Dystrophy, Type 1 (DM1) and Myotonic Dystrophy, run by Expansion Therapeutics, Inc. at 4 sites in United States.
Trial summary
Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion. The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants diagnosed with Myotonic Dystrophy, Type 1 (DM1). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
- What is being tested?
- ERX-963
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 4 sitesUnited States
- Study dates
- 17 Jun 2019to 30 Apr 2020
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 to 65 years of age
- DM1 defined by genetic testing or clinical-confirmation
- Epworth Sleepiness Scale (ESS) of > 11 or participants who have long sleep periods of an average of > 10 hours a day
- Age of onset of DM1 greater than 16 years
Who cannot join
- Significant respiratory compromise
- Significant cardiac disease
- Diagnosis of symptomatic Restless Leg Syndrome or significant untreated nocturnal hypoxias
- Significant moderate to severe hepatic insufficiency
- Clinically active depression, anxiety, or other medical condition that, in the investigator's opinion, would interfere with the safety and efficacy assessments
- History of seizures
- History of panic disorders
Trial locations and contacts
4 sites in 1 country.
United States4 sites
The Center for Sleep & Wake Disorders
Chevy Chase, Maryland, 20815
University of Iowa
Iowa City, Iowa, 52242
Sleep Medicine Specialists of South Florida
Miami, Florida, 33126
Stanford Neurosciences Health Center
Palo Alto, California, 94305
About this study
Official title: Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1
Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion.
The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants diagnosed with Myotonic Dystrophy, Type 1 (DM1).
The secondary purpose is to evaluate the potential of ERX-963 treatment to reduce excessive daytime sleepiness / hypersomnia and improve cognitive function in DM1 participants compared to placebo treatment.
Read the detailed description
This study is evaluating single administration of two dose levels of ERX-963 to explore the relationship between dose, safety, tolerability, exposure and clinical benefit. This is a multi-center, randomized, double-blind, placebo-controlled, two-treatment period crossover study in two cohorts of participants with DM1.
Participants who have consented and meet eligibility criteria will receive two treatments, placebo and ERX-963, in a randomized crossover fashion with a washout period between the treatments. On treatment days, participants will receive treatment followed by repeated blood collection for pharmacokinetic analysis and administration of a battery of outcome measures relevant to sleep and cognition.
- Being tested
- ERX-963, Placebo
- Keywords
- Excessive Daytime Sleepiness, hypersomnia
- Medical subject headings
- Myotonic Dystrophy, Disorders of Excessive Somnolence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ERX-963Drug
Active medicine
PlaceboDrug
Comparator
| Study group | Role | What they receive |
|---|---|---|
| ERX-963 then placebo | Experimental | Participants in this arm will receive ERX-963 followed by a washout period. After the washout period, participants will receive placebo. Receives: Drug: ERX-963 |
| Placebo then ERX-963 | Experimental | Participants in this arm will receive placebo followed by a washout period. After the washout period, participants will receive ERX-963. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo
An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study intervention. Treatment-emergent AEs were AEs which started between the date and time of study drug dosing and through Study Day 2, within each period. Drug-related AEs were assessed by the investigator to determine the relationship (related or unrelated) between the study intervention and each AE occurrence.
Measured: Adverse Events were collected from screening to the End of Study Visit, up to 57 days
Secondary outcomes (5)
Assess the Effect of ERX-963 on the Stanford Sleepiness Scale Score Compared to the Effect of Placebo
Participants will self-report their level of sleepiness by self-rated questionnaire "Stanford Sleepiness Scale" (SSS). This is a single item questionnaire on a 7-point scale (1-7). Higher values indicate worse outcome.
Measured: From dosing to approximately 2 hours
Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo
The PGI-I is a 7-point rating system used by the patient to rate their overall clinical condition after intervention relative to before intervention where 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse, and 7=very much worse.
Measured: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo
The CGI-I is a 7-point rating system used by the clinician or investigator to compare the patient's overall clinical condition after intervention relative to before intervention where 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse, and 7=very much worse (Guy, 1976; Busner, 2007).
Measured: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
Assess the Effect of ERX-963 on the Psychomotor Vigilance Task (PVT)
Participants will be tested for their response time and number of lapses during the PVT.
Measured: From dosing to approximately 2 hours
Assess the Effect of ERX-963 on the One-back Task
Participants will be tested for the proportion of correct response to the One-back task.
Measured: From dosing to approximately 2 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2019
- Study start17 Jun 2019
- Primary completion31 Mar 2020
- Study completion30 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myotonic Dystrophy, Type 1 (DM1)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Expansion Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elliot Ehrich, MD, Study director, Chief Medical Officer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03959189
- Sponsor's protocol ID
- ERX-963-001
- Status last verified
- May 2021
- First submitted
- 16 May 2019
- Last updated
- 23 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT03959189
- Acronym / sponsor ID
- ERX-963-001
- Keywords
- Excessive Daytime Sleepiness, hypersomnia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Expansion Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Jun 2019 (actual)
- Primary completion
- 31 Mar 2020 (actual)
- Study completion
- 30 Apr 2020 (actual)
- First posted
- 22 May 2019 (actual)
- Results
- Posted 23 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion.
The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants diagnosed with Myotonic Dystrophy, Type 1 (DM1).
The secondary purpose is to evaluate the potential of ERX-963 treatment to reduce excessive daytime sleepiness / hypersomnia and improve cognitive function in DM1 participants compared to placebo treatment.
Detailed description
This study is evaluating single administration of two dose levels of ERX-963 to explore the relationship between dose, safety, tolerability, exposure and clinical benefit. This is a multi-center, randomized, double-blind, placebo-controlled, two-treatment period crossover study in two cohorts of participants with DM1.
Participants who have consented and meet eligibility criteria will receive two treatments, placebo and ERX-963, in a randomized crossover fashion with a washout period between the treatments. On treatment days, participants will receive treatment followed by repeated blood collection for pharmacokinetic analysis and administration of a battery of outcome measures relevant to sleep and cognition.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ERX-963 then placebo | Experimental | Drug: ERX-963Participants in this arm will receive ERX-963 followed by a washout period. After the washout period, participants will receive placebo. |
| Placebo then ERX-963 | Experimental | Drug: PlaceboParticipants in this arm will receive placebo followed by a washout period. After the washout period, participants will receive ERX-963. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 18 to 65 years of age
- DM1 defined by genetic testing or clinical-confirmation
- Epworth Sleepiness Scale (ESS) of > 11 or participants who have long sleep periods of an average of > 10 hours a day
- Age of onset of DM1 greater than 16 years
Key exclusion criteria (7 listed)
- Significant respiratory compromise
- Significant cardiac disease
- Diagnosis of symptomatic Restless Leg Syndrome or significant untreated nocturnal hypoxias
- Significant moderate to severe hepatic insufficiency
- Clinically active depression, anxiety, or other medical condition that, in the investigator's opinion, would interfere with the safety and efficacy assessments
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ERX-963Active medicine | Drug | ERX-963 then placebo |
| PlaceboComparator | Drug | Placebo then ERX-963 |
Primary outcome
Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo
Time frame: Adverse Events were collected from screening to the End of Study Visit, up to 57 days
An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study intervention. Treatment-emergent AEs were AEs which started between the date and time of study drug dosing and through Study Day 2, within each period. Drug-related AEs were assessed by the investigator to determine the relationship (related or unrelated) between the study intervention and each AE occurrence.
5 secondary outcomes
Assess the Effect of ERX-963 on the Stanford Sleepiness Scale Score Compared to the Effect of Placebo
Time frame: From dosing to approximately 2 hours
Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo
Time frame: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo
Time frame: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
Assess the Effect of ERX-963 on the Psychomotor Vigilance Task (PVT)
Time frame: From dosing to approximately 2 hours
Assess the Effect of ERX-963 on the One-back Task
Time frame: From dosing to approximately 2 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Expansion Therapeutics Corporate WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elliot Ehrich, MD study director
Chief Medical Officer
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ERX-963 in people with Myotonic Dystrophy, Type 1 (DM1) and Myotonic Dystrophy, run by Expansion Therapeutics, Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo (time frame: Adverse Events were collected from screening to the End of Study Visit, up to 57 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2021.